EP3923995A4 - Künstliche expressionskonstrukte zur selektiven modulation der genexpression in ausgewählten nervenzellpopulationen - Google Patents

Künstliche expressionskonstrukte zur selektiven modulation der genexpression in ausgewählten nervenzellpopulationen Download PDF

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Publication number
EP3923995A4
EP3923995A4 EP20756018.6A EP20756018A EP3923995A4 EP 3923995 A4 EP3923995 A4 EP 3923995A4 EP 20756018 A EP20756018 A EP 20756018A EP 3923995 A4 EP3923995 A4 EP 3923995A4
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European Patent Office
Prior art keywords
cell populations
neuronal cell
modulating gene
selectively modulating
gene expression
Prior art date
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Pending
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EP20756018.6A
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English (en)
French (fr)
Other versions
EP3923995A2 (de
Inventor
Jonathan Ting
Boaz P. LEVI
Bosiljka Tasic
John K. Mich
Erik HESS
Edward Sebastian LEIN
Lucas T. GRAYBUCK
Tanya DAIGLE
Hongkui Zeng
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Allen Institute
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Allen Institute
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Publication of EP3923995A2 publication Critical patent/EP3923995A2/de
Publication of EP3923995A4 publication Critical patent/EP3923995A4/de
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2267/03—Animal model, e.g. for test or diseases
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
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EP20756018.6A 2019-02-15 2020-02-14 Künstliche expressionskonstrukte zur selektiven modulation der genexpression in ausgewählten nervenzellpopulationen Pending EP3923995A4 (de)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962806660P 2019-02-15 2019-02-15
US201962806686P 2019-02-15 2019-02-15
US201962874859P 2019-07-16 2019-07-16
PCT/US2020/018416 WO2020168279A2 (en) 2019-02-15 2020-02-14 Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations

Publications (2)

Publication Number Publication Date
EP3923995A2 EP3923995A2 (de) 2021-12-22
EP3923995A4 true EP3923995A4 (de) 2023-05-17

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EP20756018.6A Pending EP3923995A4 (de) 2019-02-15 2020-02-14 Künstliche expressionskonstrukte zur selektiven modulation der genexpression in ausgewählten nervenzellpopulationen

Country Status (6)

Country Link
US (1) US20220249703A1 (de)
EP (1) EP3923995A4 (de)
JP (1) JP2022520437A (de)
AU (1) AU2020223367A1 (de)
CA (1) CA3142948A1 (de)
WO (1) WO2020168279A2 (de)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230002788A (ko) * 2020-04-21 2023-01-05 알렌 인스티튜트 신피질 레이어 5 글루타메이트성 뉴런에서 유전자 발현을 선택적으로 조절하기 위한 인공 발현 작제물
AU2021378285A1 (en) * 2020-11-10 2023-06-22 Allen Institute Artificial expression constructs for modulating gene expression in chandelier cells
WO2022212585A1 (en) * 2021-03-30 2022-10-06 Allen Institute Artificial expression constructs for modulating gene expression in claustrum neurons
EP4413142A1 (de) * 2021-10-05 2024-08-14 Allen Institute Künstliche expressionskonstrukte zur modulation der genexpression im zerebellum und einem sekundärzellentyp

Citations (2)

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WO2017100671A1 (en) * 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
WO2020097121A1 (en) * 2018-11-05 2020-05-14 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons

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PT1071806E (pt) * 1998-04-24 2004-09-30 Univ Florida Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica
US20070037165A1 (en) * 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
EP3702460A1 (de) * 2010-11-12 2020-09-02 The General Hospital Corporation Polycombassoziierte nichtcodierende rnas
US9284575B2 (en) * 2012-03-06 2016-03-15 Duke University Synthetic regulation of gene expression
WO2013155222A2 (en) * 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
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WO2017100671A1 (en) * 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
WO2020097121A1 (en) * 2018-11-05 2020-05-14 Allen Institute Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons

Non-Patent Citations (2)

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Title
DIMIDSCHSTEIN JORDANE ET AL: "A viral strategy for targeting and manipulating interneurons across vertebrate species", NATURE NEUROSCIENCE, vol. 19, no. 12, 1 December 2016 (2016-12-01), New York, pages 1743 - 1749, XP055872737, ISSN: 1097-6256, Retrieved from the Internet <URL:https://www.nature.com/articles/nn.4430.pdf> DOI: 10.1038/nn.4430 *
GRAYBUCK LUCAS T. ET AL: "Prospective, brain-wide labeling of neuronal subclasses with enhancer-driven AAVs", BIORXIV, 20 January 2019 (2019-01-20), XP055940913, Retrieved from the Internet <URL:https://web.archive.org/web/20190430183217id_/https://www.biorxiv.org/content/biorxiv/early/2019/01/31/525014.full.pdf> [retrieved on 20220711], DOI: 10.1101/525014 *

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WO2020168279A3 (en) 2020-09-24
EP3923995A2 (de) 2021-12-22
US20220249703A1 (en) 2022-08-11
AU2020223367A1 (en) 2021-10-07
JP2022520437A (ja) 2022-03-30
CA3142948A1 (en) 2020-08-20
WO2020168279A2 (en) 2020-08-20

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